Liver drug study for rare disease pulled before it started

NCT ID NCT07123363

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled This study
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

Summary

This study aimed to see if the drug bitopertin is safe for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who also have early signs of liver disease. Researchers planned to check for side effects and measure changes in protoporphyrin levels in blood, bile, liver, and stool. However, the study was withdrawn before any participants were enrolled.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Expected to start

Jan 2026

An estimate. Start dates often move.

Expected to finish

Dec 2035

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of EEP or XLP that has been biochemically and genetically verified prior diagnosis of EPP or XLP * Age 18-75 years * Increased liver stiffness at baseline, defined as stiffness measurement \[E value\] by: Fibroscan \>7 kPa; MRE that is \>3.0 kPa; Velacur/Sonicincyte that is \>6.0 kPa; and/or elevated liver enzymes: serum ALT \> 2 X upper limit of normal (ULN), but not greater than 5 X ULN and/or serum AP \> 2 X ULN, but not greater than 5 X ULN * INR \< 1.4 * Compensated liver disease at baseline, defined as lack of clinically evident ascites, encephalopathy, hepatocellular carcinoma, clinically evident icterus or jaundice, peripheral edema. * Willingness and ability to volunteer and provide informed consent for a long-term study that will include liver biopsies and collection of bile from the second portion of the duodenum, at baseline and at the end of study. In addition, follow-up visits will be planned every 26 weeks throughout the study, with plans for repeat routine/safety labs at each visit and for measures of liver stiffness and markers of hepatic status and fibrosis (Fib-4, APRI, ELF, Fibrotest) performed once every 6 months. Exclusion Criteria: * Chronic hepatitis B, C, D, or E; * Human immunodeficiency (HIV) infection; * Alcohol use \> 14 units/week for men or \> 7 units/week for women; * Pregnancy or breast feeding among women; * Any known active malignancy other than small and localized squamous or basal cell carcinoma of the skin; * Advanced or decompensated heart, lung, kidney, liver, or neuro-psychiatric disease; * History of diagnosed depression or suicidality; * History of diagnosed substance abuse or poor impulse control; * Any other conditions that, in the opinion of the Investigator, renders the individual unfit to participate

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.